Pharma/Biotech – USA, Europe Regulatory News, Dec 2023 Vol. 1

USA

Significant Public Response to FDA’s Proposed Oversight of Laboratory-Developed Tests

The FDA has received over 2,100 comments on its proposed rule to regulate laboratory-developed tests (LDTs). Feedback varies widely, with many identical submissions opposing the rule. Supporters argue the proposal will improve test reliability, while critics warn it could increase regulatory burden and reduce access to infectious disease testing.

FDA Finalizes Rule to Improve Consumer Understanding of Drug Risks in DTC Advertising

The FDA issued a final rule emphasizing how risk information should be presented in direct-to-consumer (DTC) television and radio advertisements for prescription drugs. The rule aims to ensure consumers can better notice, process, and understand key safety information.

ICH Adopts Updated Guidance on Viral Safety Evaluation

The International Council for Harmonisation (ICH) has adopted the Q5A(R2) guideline, which updates recommendations on evaluating viral safety for biotechnology-derived products.

Senate Committee Advances PBM Reform Legislation

The Senate Finance Committee unanimously approved the Better Mental Health Care, Lower Drug Cost and Extenders Act. The bill seeks to address pharmacy benefit manager (PBM) practices that lawmakers say contribute to rising prescription drug costs.

Draft Guidance on Manufacturing Changes for Cell and Gene Therapies Raises Concerns

Industry stakeholders argue that FDA’s draft guidance on reporting manufacturing changes for cellular and gene therapy (CGT) products places excessive emphasis on clinical trials and underestimates the value of bridging studies. The guidance would require manufacturers of both investigational and approved CGTs to notify FDA of any manufacturing changes that may influence safety or efficacy.

Leveraging Collaboration and AI for Drug Repurposing

Researchers continue exploring opportunities to repurpose approved drugs for new indications. While conceptually straightforward, repurposing can be complex in practice, requiring significant data and analysis to identify and validate alternative uses.

EUROPE

EMA Broadens Review of GLP-1 Agonists Amid Concerns Over Suicidal Ideation

The EMA is expanding its investigation into reports of suicidal thoughts among users of GLP-1 receptor agonists. Manufacturers have been asked to address additional questions from regulators. Meanwhile, these weight-loss and diabetes medications continue to grow in popularity across Europe.

List of Medicines Under Additional Monitoring Updated

EMA has published an updated list of medicines subject to additional monitoring. These products display a black inverted triangle in their package leaflets and SmPCs, signaling that regulatory authorities are monitoring them closely.

Updated IRIS Guide for Applicants

EMA has revised its IRIS platform guide, providing updated instructions on how applicants can submit scientific procedures—including orphan designation, scientific advice, PRIME requests, marketing status reports, inspections, and veterinary signal management—as well as applications for parallel distribution.

Guidance on Parallel Distribution Procedures

EMA has issued updated regulatory and procedural guidance for parallel distributors, outlining how they can meet their obligations while ensuring compliance with EU legislation.

Updated EMA Post‑Authorization Procedural Guidance

EMA has updated its post-authorization procedural advice for centralized procedure users. Revisions include updated instructions for submitting Type IA/IAIN variations and clarifying how grouped variation applications are assigned procedure numbers.

Updated EMA Pre‑Authorization Procedural Guidance

EMA has issued updates to its pre‑authorization guidance, including revised instructions for submitting an Active Substance Master File (ASMF).

Guide to EMA Publications on Human Medicines

EMA has updated its guide explaining the types of information it publishes on human medicines throughout their lifecycle—from early development and authorization assessment to post-authorization changes, safety reviews, and withdrawals. The guide helps stakeholders understand what information is available, when it is published, and where it can be found.